Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy

Nanomedicine (Lond). 2017 Dec;12(23):2591-2606. doi: 10.2217/nnm-2017-0221. Epub 2017 Nov 7.


Aim: Polymersomes are created to deliver an enzyme-based therapy to the brain in lysosomal storage disease patients.

Materials & methods: Polymersomes are formed via the injection method using poly(ethylene glycol)-b-poly(lactic acid) (PEGPLA) and bound to apolipoprotein E, to create a brain-targeted delivery vehicle.

Results: Polymersomes have a smallest average diameter of 145 ± 21 nm and encapsulate β-galactosidase at 72.0 ± 12.2% efficiency. PEGPLA polymersomes demonstrate limited release at physiologic pH (7.4), with a burst release at the acidic pH (4.8) of the lysosome. PEGPLA polymersomes facilitate delivery of active β-galactosidase to an in vitro model of GM1 gangliosidosis.

Conclusion: The foundation has been laid for testing of PEGPLA polymersomes to deliver enzymatic treatments to the brain in lysosomal storage disorders for the first time.

Keywords: GM1 gangliosidosis; blood–brain barrier delivery; enzyme replacement therapy; nanomedicine; polymersomes.

MeSH terms

  • Brain / metabolism
  • Cell Line
  • Drug Carriers / chemistry*
  • Drug Liberation
  • Enzyme Replacement Therapy / methods*
  • Gangliosidosis, GM1 / drug therapy
  • Humans
  • Hydrogen-Ion Concentration
  • Lactates / chemistry*
  • Nanoparticles / chemistry
  • Particle Size
  • Permeability
  • Polyethylene Glycols / chemistry*
  • Surface Properties
  • beta-Galactosidase / pharmacology*


  • Drug Carriers
  • Lactates
  • poly(lactic acid-ethylene glycol)
  • Polyethylene Glycols
  • beta-Galactosidase